Search: (WFRF:(Bishop J. A.)) srt2:(2015-2019) > (2018) > Nusinersen versus S...
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000 | 04575naa a2200649 4500 | |
001 | oai:gup.ub.gu.se/265106 | |
003 | SwePub | |
008 | 240528s2018 | |||||||||||000 ||eng| | |
024 | 7 | a https://gup.ub.gu.se/publication/2651062 URI |
024 | 7 | a https://doi.org/10.1056/NEJMoa17105042 DOI |
040 | a (SwePub)gu | |
041 | a eng | |
042 | 9 SwePub | |
072 | 7 | a ref2 swepub-contenttype |
072 | 7 | a art2 swepub-publicationtype |
100 | 1 | a Mercuri, E.4 aut |
245 | 1 0 | a Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy |
264 | 1 | c 2018 |
520 | a BACKGROUND Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). METHODS We conducted a multicenter, double-blind, sham-controlled, phase 3 trial of nusinersen in 126 children with SMA who had symptom onset after 6 months of age. The children were randomly assigned, in a 2: 1 ratio, to undergo intrathecal administration of nusinersen at a dose of 12 mg (nusinersen group) or a sham procedure (control group) on days 1, 29, 85, and 274. The primary end point was the least-squares mean change from baseline in the Hammersmith Functional Motor Scale-Expanded (HFMSE) score at 15 months of treatment; HFMSE scores range from 0 to 66, with higher scores indicating better motor function. Secondary end points included the percentage of children with a clinically meaningful increase from baseline in the HFMSE score (>= 3 points), an outcome that indicates improvement in at least two motor skills. RESULTS In the prespecified interim analysis, there was a least-squares mean increase from baseline to month 15 in the HFMSE score in the nusinersen group (by 4.0 points) and a least-squares mean decrease in the control group (by -1.9 points), with a significant between-group difference favoring nusinersen (least-squares mean difference in change, 5.9 points; 95% confidence interval, 3.7 to 8.1; P< 0.001). This result prompted early termination of the trial. Results of the final analysis were consistent with results of the interim analysis. In the final analysis, 57% of the children in the nusinersen group as compared with 26% in the control group had an increase from baseline to month 15 in the HFMSE score of at least 3 points (P< 0.001), and the overall incidence of adverse events was similar in the nusinersen group and the control group (93% and 100%, respectively). CONCLUSIONS Among children with later-onset SMA, those who received nusinersen had significant and clinically meaningful improvement in motor function as compared with those in the control group. (Funded by Biogen and Ionis Pharmaceuticals; CHERISH ClinicalTrials. gov number, NCT02292537.) | |
650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Pediatrik0 (SwePub)302212 hsv//swe |
650 | 7 | a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Pediatrics0 (SwePub)302212 hsv//eng |
653 | a functional motor scale | |
653 | a lumbar puncture | |
653 | a headache | |
653 | a children | |
653 | a severity | |
653 | a backache | |
653 | a protein | |
653 | a trials | |
653 | a General & Internal Medicine | |
700 | 1 | a Darras, B. T.4 aut |
700 | 1 | a Chiriboga, C. A.4 aut |
700 | 1 | a Day, J. W.4 aut |
700 | 1 | a Campbell, C.4 aut |
700 | 1 | a Connolly, A. M.4 aut |
700 | 1 | a Iannaccone, S. T.4 aut |
700 | 1 | a Kirschner, J.4 aut |
700 | 1 | a Kuntz, N. L.4 aut |
700 | 1 | a Saito, K.4 aut |
700 | 1 | a Shieh, P. B.4 aut |
700 | 1 | a Tulinius, Mar,d 1953u Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics4 aut0 (Swepub:gu)xtulim |
700 | 1 | a Mazzone, E. S.4 aut |
700 | 1 | a Montes, J.4 aut |
700 | 1 | a Bishop, K. M.4 aut |
700 | 1 | a Yang, Q.4 aut |
700 | 1 | a Foster, R.4 aut |
700 | 1 | a Gheuens, S.4 aut |
700 | 1 | a Bennett, C. F.4 aut |
700 | 1 | a Farwell, W.4 aut |
700 | 1 | a Schneider, E.4 aut |
700 | 1 | a De Vivo, D. C.4 aut |
700 | 1 | a Finkel, R. S.4 aut |
700 | 1 | a Cherish Study Grp, Cherish Study Grp4 aut |
710 | 2 | a Göteborgs universitetb Institutionen för kliniska vetenskaper, Avdelningen för pediatrik4 org |
773 | 0 | t New England Journal of Medicineg 378:7, s. 625-635q 378:7<625-635x 0028-4793 |
856 | 4 8 | u https://gup.ub.gu.se/publication/265106 |
856 | 4 8 | u https://doi.org/10.1056/NEJMoa1710504 |
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